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Clinical Trial Report

Gene Therapy Trial Report

Summary

Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)

NCTID NCT06467344 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Disease Ontology Term DOID:0050817
Compound Name ACDN-01
Sponsor Ascidian Therapeutics, Inc
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 13
Results Posted Not Available

Therapy Information

Target Gene/Variant ABCA4 pre-mRNA
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Exon skipping/splice editor
Route of Administration Subretinal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2024-05-31
Completion Date 2030-12-01
Last Update 2025-01-22

Participation Criteria

Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 10
Locations United States

Regulatory Information

Has US IND True
FDA Designations Fast Track, Rare Pediatric Disease Designation
Recent Updates Rare Pediatric Disease designation granted 4/25/24

Resources/Links

Clinical Publications
Preclinical Publications